Raising the bar for using surrogate endpoints in drug regulation and health technology assessment
journal contribution
posted on 2021-11-12, 16:08 authored by Dalia Dawoud, Huseyin Naci, Oriana Ciani, Sylwia BujkiewiczIn June 2021, the US Food and Drug Administration
granted accelerated approval to aducanumab for
treating Alzheimer’s disease based on the drug’s
amyloid reducing effects. This was despite evidence
from several earlier studies that shrinkage of
β-amyloid protein plaques does not predictably delay
cognitive impairment.1 The controversial decision
has drawn attention to the use of surrogate
endpoints—laboratory values, radiographic images,
or other physical measures that may serve as
indicators of clinical outcomes such as symptom
control or mortality—in clinical trials of new drugs.2
In fact, the approval of aducanumab is only the latest
example of growing regulatory reliance on surrogate
endpoints, even though their use can cause problems
for patients, clinicians, drug regulators, and health
technology assessment bodies.
We argue for more selective use of surrogate
endpoints when evaluating new drugs, restricting
their use to chronic diseases, especially when
collecting data on patient relevant clinical outcomes
requires trials with unattainably long follow up.
History
Citation
BMJ 2021; 374 doi: https://doi.org/10.1136/bmj.n2191 (Published 16 September 2021) Cite this as: BMJ 2021;374:n2191Author affiliation
Department of Health Sciences, University of LeicesterVersion
- AM (Accepted Manuscript)
Published in
BRITISH MEDICAL JOURNALVolume
374Publisher
BMJ Publishing Groupissn
1759-2151eissn
0959-8138Copyright date
2021Available date
2021-11-12Publisher DOI
Language
EnglishPublisher version
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